Kornimate/medical-research-clean huggingface dataset:
a phase 3 study of ocu400 gene therapy for the treatment of retinitis pigmentosa

Conditions: Retinitis Pigmentosa

Summary: this is a phase 3 study to assess the efficacy, safety and tolerability of ocu400 in patients with retinitis pigmentosa (rp) associated with rho mutations and patients with any other rp associated mutation with a clinical phenotype of rp. this is a multicenter, assessor blinded and randomized study which will enroll 150 subjects.

a total of one hundred and fifty (150) rp participants will be enrolled in this study- 75 in each of either two arms, rho arm (n=75) or gene agnostic arm (n=75). rho arm will only enroll participants with confirmed genetic diagnosis of mutation in rho gene; whereas gene agnostic arm will enroll rp participants based on clinical diagnosis of rp and a confirmed genetic diagnosis with a gene associated with rp. subjects in each arm will be randomized into treatment (n=50) and control groups (n=25). subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of ocu400 if both eyes meet inclusion criteria. control or untreated group subjects will receive ocu400 subretinal injection after completion of 12-month follow-up.

Eligibility criteria:
inclusion criteria: 1. males or females ≥ 5 years of age 2. clinical diagnosis of rp with confirmed genetic diagnosis of autosomal dominant rho mutation 3. clinical diagnosis of syndromic or non-syndromic rp with/without confirmed genetic diagnosis of any other rp associated mutation (except ad-nr2e3) 4. bcva ≤ 75 letters and ≥25 letters as measured by an etdrs chart 5. visual field of \>5° in any meridian as measured by a iii4e isopter or equivalent 6. able to perform a luminance ldna at certain light intensity at the screening visit 7. presence of photoreceptors as determined by sd-oct exclusion criteria: 1. subject lacks evidence of outer nuclear layer 2. previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year. 3. history of any corticosteroid contraindication, corticosteroid related iop spikes or uncontrolled glaucoma. 4. cataract surgery within 3 months. yag capsulotomy within 1 month. any other intraocular surgery within 6 months. 5. active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or current retinal detachment or retinal implant. 6. breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Keywords: modified gene therapy retinitis pigmentosa rho rp nr2e3 subretinal injection
neuropathic pain study with botulinum toxin a in spinal cord injury patients

Conditions: Spinal Cord Injury; Neuropathic Pain

Summary: the purpose of this study is to determine whether botulinum toxin a is effective in the treatment of neuropathic pain in spinal cord injury patients.

neuropathic pain remains a significant cause of life quality deterioration. this study includes spinal cord injury patients with refractory chronic neuropathic pain and investigates whether injection with botulinum toxin a improves pain scores.

Eligibility criteria:
inclusion criteria: * more than twenty years of age * paraplegic or tetraplegic due to cervical and thoracic spinal cord injury * more than 12 months since spinal cord injury and asia impairment scale unchanged for more than 6 months * persistence of neuropathic pain for more than three months or remission and recurrence of neuropathic pain for more than six months * a pain score of 40mm or more on the visual analogue scale exclusion criteria: * neuropathic pain caused by confounding factors other than spinal cord injury * contraindicated for botulinum toxin type a * a change in pain medication one month prior to study enrollment * a condition involving neuromuscular junction (ex. eaton lambert disease, myasthenia gravis) * person who received botulinum toxin type a within three months prior to study enrollment
value of ultrasound and computed tomography in the diagnosis of acute appendicitis with histopathology as gold standard.

Conditions: Appendicitis Acute

Summary: this study is a retrospective evaluation of all patients treated surgically for appendicitis over a period of 3 years at the sint- andries hospital, tielt. belgium. the goal is to evaluate the value of ultrasound (us), computed tomography (ct) and laboratory testing in diagnosing acute appendicitis and to look for the possible impact on the treatment.


Eligibility criteria:
inclusion criteria: * suspicion of acute appendicitis exclusion criteria: * none

Keywords: appendicitis general surgery ultrasound computed tomography
endoscopic band ligation (ebl) versus propranolol for primary prophylaxis of variceal bleeding

Conditions: Variceal Bleeding; Cirrhosis

Summary: this study is performed to compare the efficacy and safety of ebl, propranolol, and ebl combined with propranolol in patients with medium or large varices.

current guidelines recommend prophylactic treatment with propranolol or endoscopic band ligation (ebl) to prevent variceal bleeding in patients with medium or large varices. however, it is unclear which treatment is more useful in regard to prevention of variceal bleeding as well as safety. in addition, the efficacy and safety of the combination of ebl and propranolol is not still defined. this study is performed to compare the efficacy and safety of ebl, propranolol, and ebl combined with propranolol in patients with medium or large varices.

Eligibility criteria:
inclusion criteria: * liver cirrhosis * age between 18 and 70 years * esophageal varices with high bleeding risk: more than f2 and red color sign * no previous history of upper gastrointestinal bleeding * no previous history of endoscopic, radiologic, or surgical therapy for varices or ascites * do not take beta-blocker, ace inhibitor, or nitrate * child-pugh score \<12 exclusion criteria: * patients with systolic blood pressure \<100 mmhg or basal heart rate \<60/min * portal vein thrombosis * uncontrolled ascites or hepatic encephalopathy * severe coagulation disorder: prothrombin time \ 1.7) or platelet count \<30,000/mm3 * medium or large sized gastric or duodenal varices * coexisting malignancy * severe cardiovascular disorder, renal failure, peritonitis, sepsis * severe erosive esophagitis, severe esophageal stricture, active gastric or duodenal ulcer * contraindication to beta-blocker * pregnancy * refusal to give consent to participate in the trial

Keywords: cirrhosis esophagus disorders varicose veins
transcranial alternating current stimulation (tacs) in patients with ataxia

Conditions: Ataxia; Spinocerebellar Ataxias; Multiple System Atrophy; Ataxia With Oculomotor Apraxia; CANVAS

Summary: the aim of the study is to evaluate the effects on motor and cognitive performance of transcranial alternating current stimulation (tacs) compared to transcranial direct current stimulation (tdcs) and placebo stimulation (sham) in patients with neurodegenerative ataxia to identify a possible rehabilitation protocol.


Eligibility criteria:
inclusion criteria: * patients with a cerebellar syndrome and quantifiable cerebellar atrophy exclusion criteria: * severe head trauma in the past * history of seizures * history of ischemic stroke or hemorrhage * pacemaker * metal implants in the head/neck region * severe comorbidity * intake of illegal drugs * pregnancy

Keywords: transcranial direct current stimulation transcranial alternate current stimulation spinocerebellar ataxia cerebellar ataxia
a study to investigate ataciguat for slowing the progression of moderate calcific aortic valve stenosis

Conditions: Moderate Aortic Valve Stenosis

Summary: the purpose of this study is to evaluate if ataciguat slows the progression of moderate calcific aortic valve stenosis in adults.

this study will be conducted in two parts: part a and part b. the purpose of part a is to investigate whether ataciguat slows the progression of aortic valve calcium (avc) deposition and whether the change in avc effects peak oxygen consumption (pvo2) in adults with moderate calcific aortic valve stenosis (cavs). the safety, tolerability, and pharmacokinetics of ataciguat will also be evaluated. the purpose of part b is to evaluate the effect of ataciguat on peak vo2 and to investigate whether ataciguat reduces the rate of aortic valve area (ava) in participants with moderate cavs. the safety and tolerability of ataciguat will also be evaluated.

Eligibility criteria:
key inclusion criteria: 1. adult male or female at least 50 years of age 2. has moderate cavs as defined by: 1. an ava of ≥1 cm2 to ≤1.50 cm2 2. an avc score between ≥600 to 1200 agatston units (au) for women and between ≥600 to 2000 au for men 3. has a left ventricular ejection fraction (ef) of ≥45% at the time of screening as determined by the echocardiography core laboratory 4. can perform cardiopulmonary exercise testing (cpet) key exclusion criteria: 1. has had a prior aortic valve replacement, repair, surgery, or intervention 2. has moderate, moderate-to-severe, or severe (grade 2 or higher) mitral stenosis, mitral regurgitation, and/or aortic regurgitation 3. has known congenital aortic valve disease including bicuspid aortic valve 4. new york heart association (nyha) class iii or class iv 5. has heart failure such as cardiomyopathy, congenital heart disease, myocarditis 6. has coronary artery disease or anticipating coronary stenting surgery 7. abnormal electrocardiogram (ecg) results or long-standing persistent or permanent atrial fibrillation

Keywords: ataciguat sgc activator sgc stimulator aortic valve stenosis moderate aortic valve stenosis calcific aortic valve stenosis cavs moderate calcific aortic valve stenosis aortic stenosis (as)
goals of care communication

Conditions: Heart Failure

Summary: this study proposes to evaluate, among adults with advanced heart failure (hf) and their health care providers, the effects of a communication intervention designed to improve patient-provider communication about goals of care (goc). patient activated goc conversations can result in providers' better understanding their patients' preferences for end-of-life care. goc conversations that occur over time may allow patients and providers to consider a wider range of options earlier in the hf illness trajectory. preliminary data suggests that the proposed goc intervention increases the occurrence and quality of patient-provider communication about end-of-life care for advanced copd. the proposed study will test the effects of the goc intervention compared to usual care, using a randomized, 2-group (n = 80, n = 40/arm), repeated-measures design. measures will be taken at a patient's baseline study entry and after the next scheduled clinic visit. specific aim 1 is to evaluate change in number of goc conversations between patient and provider between the two groups. specific aim 2 is to examine whether the intervention increases patient quality of life, referrals to palliative care, advance care planning, decreases depression and anxiety. specific aim 3 is to describe the feasibility, acceptability, perceived benefits, burden, and implementation success of the intervention in the heart failure clinic setting. patients will be randomized to the goc intervention or to receive usual care. patients in the goc intervention group, telephone coaching by a research nurse to help patients role-play talking with their hf providers. both patients and their providers, will be provided with summaries of individualized, patient-centered information about patient preferences for communicating about end-of-life care and patient self-identified barriers to and facilitators of communication about end-of-life care with tips on how to initiate goals of care discussions. providers of patients in the intervention group will also be provided with patient-specific prognostic information based on the seattle heart failure model. the goc intervention will be tested in a real-world clinic setting. this intervention is expected to be relatively cost-effective and easily translated into general cardiology practice in the future. the study is expected to make significant contributions to provision of earlier access to palliative care in the advanced hf disease trajectory.


Eligibility criteria:
inclusion criteria: 1. diagnosis of hf from either systolic or diastolic dysfunction, with an ejection fraction of ≤ 40% 2. completion of an outpatient hf visit within the past 6 months 3. ability to read, write, and speak in english exclusion criteria: 1. short blessed cognitive score \> 10 to rule out significant cognitive impairment;82 2. diagnosis of any additional terminal illness with life expectancy of ≤ 1 year not related to heart disease 3. psychiatric illness that required hospitalization in the past year; and (4) age less than 18 years
evaluation of macular structural and functional function following extensive retinal laser therapy in patients with proliferative diabetic retinopathy

Conditions: Diabetes Mellitus, Type 2

Summary: the present study is a semi-experimental clinical trial that 20 patients (40 eyes) with pdr underwent prp with arc with a spot number of 1,200 to 1,500 per eye and spot size 500 micron with a duration of 200 ms. the variables of contrast sensitivity, macula thickness under fovea , bcva, the width of thyroid under fovea , rnfl thickness in the four areas around the disc, and the quality of life questionnaire of all patients were recorded at the beginning of the study.retinal and choroid under fovea thickness and rnfl thickness were assessed in four areas, one week and one month after the end of treatment, and all the variables mentioned earlier were evaluated three months after prp surgery. this study is single group that the outcomes are compared before and after the intervention


Eligibility criteria:
inclusion criteria: * diabetes mellitus, type 2 with pdr * having conscious satisfaction exclusion criteria: * patients with a history of other eye diseases * patients with a history of previous laser treatment * patients with a history of injection of anti-vgef * patients with vitreous hemorrhage * patients with macula edema * patients with myopia more than -3 diopters or hyperopiamore than +3 diopters
written exposure therapy and app-delivered mindfulness meditation for ptsd in china: a pilot study

Conditions: PTSD; Insomnia, Secondary

Summary: the goal of this study is to use a pilot rct to assess the effectiveness of written exposure therapy (wet) and a mindfulness-based app (mba) for managing ptsd and comorbid insomnia in china. the main questions it aims to answer are: 1. does wet alone reduce ptsd symptoms in chinese patients with ptsd and subthreshold ptsd? 2. does the addition of mba to wet lead to greater reductions in comorbid insomnia symptoms compared to wet alone? researchers will compare wet alone and mcc to see if wet provides benefits in managing ptsd. researchers will compare wet plus mba and wet group to see if the integrated mba treatment provides additional benefits in managing insomnia. participants will: * undergo random assignment to one of three groups: wet, wet plus mba, or mcc. * receive clinical interviews for primary outcomes (ptsd symptoms) at baseline, posttreatment, and during follow-ups. * provide self-reported data on ptsd symptoms and insomnia severity at multiple time points. * in the wet plus mba group, additionally use a mindfulness-based app as part of their treatment.


Eligibility criteria:
inclusion criteria: 1. adult male and female aged between 18-65 years old; 2. a diagnosis of ptsd or subthreshold ptsd (ptsd symptom scale, interview version for dsm-5, pssi-5); 3. if currently taking psychotropic medication, taking a stable one for at least 4 weeks; 4. a smartphone owner and no obstacle to the now meditation app; 5. scoring 12 or more on insomnia severity index (isi; the symptoms appear after the diagnosis of full/subthreshold ptsd). exclusion criteria: 1. a diagnosis of bipolar disorder or psychotic disorder; 2. current substance dependence; 3. evidence of a moderate or severe traumatic brain injury (as determined by the obstacle to comprehending the baseline screening questionnaires); 4. serious suicidal ideation (as determined by the scale for suicidal ideation); 5. other psychiatric disorders severe enough to warrant designation as the primary disorder; 6. taking psychotherapy for ptsd currently.
interprofessional group intervention to enhance compassion satisfaction and resilience

Conditions: Compassion Fatigue; Resilience

Summary: the purpose of this study is to evaluate an educational professional development program designed to assist health care professionals in developing self-awareness and self-care choices as a means to avoid compassion fatigue and improve resilience.

professional caregivers are at risk of compassion fatigue due to the nature of their work and repeated exposure over time to work-related stressors. symptoms of compassion fatigue may include decreased concentration/productivity, increased sick days, and high turnover rates which directly effect patient satisfaction and safety. lack of data that supports this use of program, though anecdotally, it has been endorsed and benefits from its use are described. quality of patient care, workforce engagement, and financial effects from turnover of staff are viewed as negative impacts from compassion fatigue. our study seeks to extend the body of knowledge with regard to use of a particular resilience program that has shown some promise in a small pilot study.

Eligibility criteria:
inclusion criteria: * employment within inpatient or outpatient setting in non-profit healthcare system as a healthcare professional * professional direct caregivers (nurses, physicians, advanced practice providers, social workers, counselors, chaplains, respiratory therapists, pharmacists exclusion criteria: * actively participating in or plans to participate in any other formalized mindfulness-based stress reduction program during the time period of the study

Keywords: compassion fatigue resilience
priority-connect 2 pilot trial

Conditions: Liver Cancer; Pancreas Cancer; Oesophageal Cancer; Gastric Cancer; Colorectal Cancer

Summary: the provision of preoperative interventions (prehabilitation: including exercise, nutrition, and psychological treatment) have been reported to reduce postoperative complications by as much as 50% and reduce hospital stay by up to 4 days compared to standard of care. postoperative multimodal interventions are likely to further benefit patients facing new challenges (e.g. stoma care), and reduce post discharge complications. therefore the virtual multimodal hub of priority-connect 2 pilot trial aims to primarily; determine the feasibility of incorporating a virtual multimodal program into the preoperative and postoperative period for patients undergoing gastrointestinal cancer surgery, the acceptability to patients, clinicians and carers of the virtual multimodal program and the acceptability to patients of being randomised to the virtual multimodal program or usual care. the secondary aim is to obtain pilot data on the likely difference in key outcomes (30 days postoperative complications, quality of life, days at home and alive at 30 days - dah30, implementation outcomes and cost outcomes) to inform the development of a substantive randomised clinical trial.


Eligibility criteria:
inclusion criteria: * adults aged ≥18 years undergoing major gastrointestinal elective surgery, including liver, pancreas, oesophagus, gastric and colorectal cancer resections with curative intent * consulting with a gastrointestinal cancer surgeon at least 1 week prior to scheduled surgery exclusion criteria: * cognitive impairment such that they are unable to provide informed consent * no access to a smart device (including mobile phone, tablet, laptop or desk computer with camera) or no internet connection

Keywords: prehabilitation gastrointestinal cancer surgery surgical outcomes randomised controlled trial
study of ds-8201a in subjects with advanced solid malignant tumors

Conditions: Advanced Solid Tumors

Summary: this is an open-label, two-part, multicenter study to evaluate the safety and tolerability of ds-8201a in participants with advanced solid malignant tumors.


Eligibility criteria:
inclusion criteria: * eastern cooperative oncology group performance status( ps) of 0 or 1. * left ventricular ejection fraction (lvef) ≥ 50% part 1: * advanced/unresectable or metastatic breast cancer or gastric or gastroesophageal junction adenocarcinoma that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. part 2a: * advanced breast cancer with her2 overexpression that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. * treated with ado-trastuzumab emtansine (t-dm1) part 2b: * advanced gastric or gastroesophageal junction adenocarcinoma with her2 overexpression that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. * treated with trastuzumab part 2c: * advanced breast cancer with her2 low expression that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. part 2d: * satisfy at least one of the following criteria 1. advanced/unresectable or metastatic solid malignant tumor with her2 expression other than breast cancer and gastric or gastroesophageal junction adenocarcinoma that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. 2. advanced/unresectable or metastatic tumor with her2 mutation that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. part 2e: * advanced breast cancer with her2 overexpression that is refractory to or intolerable with standard treatment, or for which no standard treatment is available. * treated with ado-trastuzumab emtansine (t-dm1) (patients with her2 overexpression only) exclusion criteria: * has a medical history of symptomatic congestive heart failure (chf) (nyha classes ii-iv) or serious cardiac arrhythmia. * has a medical history of myocardial infarction or unstable angina. * has a qtc prolongation to \> 450 millisecond (ms) in males and \> 470 ms in females. * has a medical history of clinically significant lung diseases

Keywords: advanced solid tumors phase 1 oncology her2 antibody drug conjugate (adc)
lipid efficacy and safety in participants with mixed hyperlipidemia (mk-0524b-024)

Conditions: Mixed Hyperlipidemia

Summary: this is a 12-week clinical trial in participants with mixed hyperlipidemia to study the effects of mk-0524b on lipids.the primary hypothesis is that mk-0524b (dosed as mk-0524a coadministered with simvastatin) will be superior to atorvastatin on decreasing the low denisity lipoprotein cholesterol (ldl-c)/high-density lipoprotein cholesterol (hdl-c) ratio for the following dose comparisons: 2g/20 mg mk-0524b versus 10 mg atorvastatin, 2g/40 mg mk-0524b versus 20 mg atorvastatin, 2g/40 mg mk-0524b versus 40 mg atorvastatin, and 2g/40 mg mk-0524b versus 80 mg atorvastatin.


Eligibility criteria:
inclusion criteria: * participant 18 to 80 years of age with mixed hyperlipidemia with ldl-c between 130 and 190 mg/dl and triglycerides between 150 and 500 mg/dl exclusion criteria: * pregnant or lactating women, or women intending to become pregnant * diabetes mellitus that is poorly controlled, newly diagnosed, or taking new or recently adjusted antidiabetic therapy (with the exception of ± 10 units of insulin) * human immunodeficiency virus (hiv) positive * any of the following within the past 3 months: heart attack, stoke, heart bypass surgery, unstable angina, angioplasty * active or chronic liver disease